{"id":"W4312065791","doi":"10.1016/j.gim.2022.10.010","title":"Safety and efficacy of avalglucosidase alfa in individuals with infantile-onset Pompe disease enrolled in the phase 2, open-label Mini-COMET study: The 6-month primary analysis report","year":2022,"lang":"en","type":"article","venue":"Genetics in Medicine","topic":"Lysosomal Storage Disorders Research","field":"Medicine","cited_by":52,"is_retracted":false,"has_abstract":true,"ca_institutions":"Sanofi (Canada)","funders":"Sanofi","keywords":"Medicine; Cohort; Adverse effect; Pediatrics; Internal medicine","routes":{"ca_aff":true,"ca_fund":false,"ca_venue":false,"about_ca":false,"invisible_to_affiliation_only":false},"retraction":null,"screen":null,"direct_labels":[],"prediction":{"model_version":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.003728435,0.0004625379,0.0009251891,0.0001940011,0.000370436,0.0005338127,0.0002400951,0.0005733201,0.00162743],"category_scores_gemma":[0.002075132,0.0002062511,0.001247316,0.0002035255,0.0002408042,0.0004842514,0.000230086,0.0007808272,0.0003039052],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0003892464,"about_ca_system_score_gemma":0.00053354,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.000353489,"about_ca_topic_score_gemma":0.0007055175,"domain_scores_codex":[0.9990996,0.0004504207,0.00006658001,0.0001440166,0.0001627595,0.00007662195],"domain_scores_gemma":[0.9984653,0.0003209482,0.0006496458,0.0001348457,0.0001857716,0.0002435003],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"randomized_trial","study_design_gemma":"nonrandomized_trial","study_design_scores_codex":[0.4932516,0.009194619,0.3619119,0.001090395,0.004806235,0.0006088715,0.000352414,0.001420319,0.02178427,0.0001720831,0.005253763,0.1001535],"study_design_scores_gemma":[0.07757131,0.2000376,0.7035214,0.0001579292,0.002241946,0.001600661,0.000108392,0.002429186,0.006742416,0.0002579783,0.005269151,0.00006212569],"study_design_candidate":"nonrandomized_trial","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9946767,0.00189908,0.0004787738,0.0001366052,0.00002614731,0.0004387975,0.00149524,0.00002962025,0.0008189875],"genre_scores_gemma":[0.9958577,0.0002703363,0.00068825,0.000260861,0.00005196405,0.0003602667,0.00214502,0.000005883871,0.0003597262],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.003728435,"threshold_uncertainty_score":0.01971811,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.04118507425872485,"score_gpt":0.3782173518220325,"score_spread":0.3370322775633076,"validation_status":"score_only:v0-immature-baseline","note":"Baseline scores from an immature model (maturity gate not passed). Scores rank; they never assert a category."}}